Vertex’s cystic fibrosis drugs have promising trial

2-drug treatment effective in trial

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Results of a clinical trial released Tuesday showed a combination of two cystic fibrosis drugs developed by Boston’s Vertex Pharmaceuticals Inc. helped people with the deadly disease breathe more easily, raising hopes the treatment could be on the market by late next year and sending the company’s stock soaring.

The clinical study of more than 1,100 cystic fibrosis patients involved a drug regimen that can treat nearly half of those suffering from the genetic disease — a huge advance compared with the 4 percent able to take a Vertex drug already on the market.

“This is life-changing,” said Aaron C. Stocks, 28, of Frederick, Md., who participated in the testing. “About three weeks after I began taking this medicine, I went for a run and the difference was unbelievable. I wasn’t winded, I wasn’t tired, I just wanted to keep going.”

Stocks, who is a case manager at the Cystic Fibrosis Foundation in Bethesda, Md. — which has helped bankroll the Vertex research program — said he and his wife hope to have a family. Until now, he said, “I never thought that far ahead.”

Vertex chief executive Jeffrey Leiden said the encouraging study results mean the company probably will file for approval of the treatment in the United States and Europe by the end of the year, and it will seek priority review by regulators. The medicine combines a variation of Vertex’s existing cystic fibrosis drug Kalydeco with its experimental compound lumacaftor.

“It’s going to potentially allow us to get a medicine to 22,000 patients around the world with the most common form of CF,” Leiden said. “And it will be the first medicine that treats the underlying cause of the disease for those patients. Every improvement in lung function, every day they stay out of the hospital, every pound they gain, is meaningful.”

Cystic fibrosis is an inherited disease that causes mucus to thicken and block the passage of air to the lungs. That makes it harder for patients to breathe and to digest food. About half of those born with the disease don’t live past 30, according to the Cystic Fibrosis Foundation.

The trial results were gratifying for Boston businessman Joe O’Donnell, who with his wife, Kathy, have been active fund-raisers for cystic fibrosis research since their son Joey died of the disease at age 12 in 1986.

“My hair was standing up on my arms all morning when I heard about this,” O’Donnell said. “This is a milestone day. It’s not the culmination. We’re in the seventh inning or the eighth inning. We don’t have FDA approval, but we will get that. We fully expect the lives of these kids are going to be transformed.”

O’Donnell, chairman of the food services company Centerplate Inc., was a pioneer in venture philanthropy, raising money to bankroll private researchers who study rare diseases. He and his wife have helped generate more than $600 million for cystic fibrosis research in the past three decades, some of which was invested in Vertex’s drug discovery work.

Tuesday’s clinical data lifted the price of Vertex stock, sending shares up 40.4 percent to $93.53 on the Nasdaq exchange. The company, which had initial success with an injectable drug to treat hepatitis C, was forced to abandon that market and shift its focus to cystic fibrosis treatments after a new generation of more effective hepatitis C pills made by competitors were approved. But its stock price fell earlier this month after some analysts expressed doubts about Vertex’s prospects for success in its cystic fibrosis trials.

That sentiment has now changed. Mark Schoenebaum, biotechnology analyst for financial firm ISI Group in New York, estimated that likely regulatory approval of the two-drug cocktail for cystic fibrosis put Vertex on track to eventually reach annual sales of as much as $5.5 billion.

“The data are good and will clearly support worldwide regulatory approval for this drug regimen,” Schoenebaum wrote in a note to investors. “I can’t see any reason why the vast majority of patients with the appropriate mutation will not want to take this regimen.”

Dr. Michael P. Boyle, director of the adult cystic fibrosis program at Johns Hopkins Hospital in Baltimore and one of the principal US investigators for the clinical trial, said the results were better than expected — not only in improving lung function but in dropping the rate of pulmonary exacerbations that can result in the hospitalization of patients.

“As a clinician and as a researcher, I was extremely excited to see the effect of this treatment not only on patients’ lung function but on [reducing] the frequency with which they become sick,” Boyle said. “We knew that if we were going to make a difference in cystic fibrosis, we had to be able to help people with the most common form of this disease.”

Kalydeco, the cystic fibrosis drug Vertex now sells, targets the cause of the condition in only 4 percent of the people suffering from the disease, about 2,000 people. It helps activate a protein that already sits on the surface of cells for those patients, enabling it to control salt and water balance on the lining of the lungs.

For the larger group of patients targeted by the two-drug cocktail, the cystic fibrosis protein is not folded correctly and sits in the center of the cells. The drugs send the protein to the surface and turn it on, alleviating
the disease’s symptoms.

“We were hopeful, but we knew this would be a harder problem to solve,” Boyle said.

Vertex is working to expand the subsets of cystic fibrosis patients that its drugs can treat.

In addition to the two-drug combination, which could eventually treat 22,000 to 30,000 patients, the company is also developing a three-drug cocktail that could treat another 17,000 people.